Speaker
Emily O’Brien, PhD, FAHA
Associate Professor
Duke Clinical Research Institute
Duke University School of Medicine
Department of Population Health Sciences
Keywords
Pragmatic Trials; Best Practices; PCORnet; Evidence-Based Practices
Key Points
- Historically, the healthcare industry has been limited by an insufficient body of evidence driving everyday clinical decision-making. Roughly a decade ago, pragmatic clinical trials (PCTs) began to gain traction as a promising solution.
- There are several advantages of PCTs. They can be embedded within healthcare systems without disrupting the clinical workflow; answer questions of major public health importance; streamline procedures and infrastructure by making use of existing data; and include diverse, representative study populations for highly generalizable results.
- But a recent analysis of clinical research site challenges noted that protocol complexity, site workload, and patient burden have increased since 2015. Though the analysis was not specific to pragmatic trials, a fundamental shift in how researchers think about study design is required across the clinical trials space.
- Additionally, evidence-based practices – even those that have been stress-tested in PCTs – are not always adopted by health systems. Trial success does not necessarily coincide with system priorities; different audiences, i.e. systems and funders, require different kinds of evidence; and 5- to 10-year studies are misaligned with systems’ 2- to 3-year decision horizons.
- The NIH Pragmatic Trials Collaboratory philosophy holds that fumbles are part of the game; we can’t improve if we only share wins, and transparency and teamwork has helped this community iterate and improve. Accordingly, the PCORnet team developed “The Playbook,” inspired by the NIH Collaboratory’s Living Textbook, as a tool for sharing and refining the best approach to national-scale research.
- The Playbook contains practical “drills” for avoiding common fumbles in recruitment, workflow, and outcome capture, and was created using a user-centered design process. They engaged PCORnet groups, partners, and members of the Playbook’s intended audience to inform and guide the content.
- Modules 1 – 5 of the Playbook, launching this year, will provide an introduction to the network. They include sections on getting started with PCORnet, utilizing the network’s resources, dissemination and implementation expectations for PCORnet studies, and case studies.
- In the long-term, the PCORnet team plans to actively review, maintain, and expand the Playbook. Additional modules are in process and targeted for release in 2026.
Discussion Themes
The success of the Playbook may depend on the willingness of investigators to share both their “best plays” and their mistakes. Dr. O’Brien was optimistic that research teams will buy into this philosophy and acknowledge it as an important piece of the evidence generation process.
The case studies that the team selected serve to illustrate A) that PCORnet trials are unique, innovative, and approaching challenges in a thoughtful, inspiring way and B) the many ways to engage with the network.